DNA, Immune Response and Packaging - Drug Discovery Digest

Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

OCTOBER 12, 2023

Can they deliver healing genes without triggering an overactive immune response? The details are disturbingly reminiscent of the famous case of Jesse Gelsinger , who died from a ferocious immune response to experimental gene therapy in September 1999. million DNA bases. Muscles stop working.

Therapies

Therapies Immune Response DNA Virus

Unveiling neoantigen-directed cancer treatment

Drug Target Review

JUNE 9, 2023

Within the collaboration, Adaptive is responsible for end-to-end preclinical TCR discovery. As part of the TCR discovery process and preclinical data package, Adaptive performs rigorous functional characterisation and safety assessments of potent, therapeutic grade antigen-specific TCRs.

Treatment

Treatment Packaging Clinical Research Therapies

A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

JUNE 27, 2024

Unlike gene editing, this “epigenetic” editing does not modify the underlying DNA sequence, but it should switch the gene off permanently, which means that this could be a one-time treatment. Like Cas9, ZFPs can serve as guide proteins to direct the tool to a target site in DNA.

Disease

Disease Therapies Engineering Clinical Trials

Codon Digest: Seeing Colors After Gene Therapy

Codon

JULY 27, 2023

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. billion units of DNA code are transcribed into more than a hundred volumes, each a thousand pages long, in type so small as to be barely legible.” a lower risk of immune responses.

Therapies

Therapies RNA DNA Engineering

Codon Digest: Seeing Colors After Gene Therapy

Codon

JULY 27, 2023

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. billion units of DNA code are transcribed into more than a hundred volumes, each a thousand pages long, in type so small as to be barely legible.” a lower risk of immune responses.

Therapies

Therapies RNA DNA Engineering

Article FDA Thank You Draft guidance on potency assays for CGT products garners extensive stakeholder input

Agency IQ

APRIL 12, 2024

For therapeutic protein products , a critical use of assays is the detection and assessment of potential anti-drug antibody (ADA) formation, signaling a product’s potential to induce an unwanted immune response. Additionally, six of these comments came from the same organization (Integrated DNA Technologies).

Production

Production FDA Therapies Biochemical Assays

Lilly Delivers Strong Second-Quarter 2021 Financial Results, Updates 2021 Financial Guidance

The Pharma Data

AUGUST 3, 2021

The company confirmed the tirzepatide SURPASS program has met global regulatory submission requirements for evaluating cardiovascular risk and its intention to submit the registration package to regulatory authorities by the end of 2021. Loxo Oncology at Lilly and Kumquat Biosciences Inc.

Production

Production Marketing Research DNA

Scaling Phage Therapy

Codon

FEBRUARY 13, 2024

Such contaminants could have the same toxic effects as the bacterial infection, or provoke a severe immune response in the patient, and therefore the phage mixtures must go through extensive purification and testing procedures. This process would begin by taking a sample of bacteria from the patient and sequencing its DNA.

Therapies

Therapies DNA Virus Clinical Trials

Tonix Pharmaceuticals Enrolls First Participant in the PRECISION Study, an Observational Study to Facilitate Development of Precision Medicine Techniques for COVID-19 Vaccines and Therapeutics

The Pharma Data

OCTOBER 14, 2020

(Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the observational PRECISION study (TNX-C002), to examine the immune responses to COVID-19 in healthy volunteers who have recovered from COVID-19 or were asymptomatic.

Vaccine

Vaccine Pharmaceuticals Small Molecule Virus

Synthetic Origins

Codon

APRIL 2, 2024

in physics at Princeton University, he had a remarkable idea: What if it were possible to build a circuit out of DNA, rather than electronics, and use it to “program” a living cell? So we engineered a system that allows the cell to package up RNAs and then secrete them in extracellular vesicles.

RNA

RNA Engineering Molecular Biology Synthetic Chemistry

FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). It aims to correct the faulty instructions at the DNA level, straightforward, at least conceptually, because mutations are deletions, of part or all of the gene. That’s not the case for ALS.

Therapies

Therapies FDA Clinical Trials DNA

Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon

APRIL 23, 2023

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain.

Vaccine

Vaccine DNA Virus Engineering

Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon

APRIL 23, 2023

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain.

Vaccine

Vaccine DNA Virus Engineering

Drug Discovery Digest

Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

Unveiling neoantigen-directed cancer treatment

Trending Sources

A therapy candidate for fatal prion diseases turns off disease-causing gene

Codon Digest: Seeing Colors After Gene Therapy

Codon Digest: Seeing Colors After Gene Therapy

Article FDA Thank You Draft guidance on potency assays for CGT products garners extensive stakeholder input

Lilly Delivers Strong Second-Quarter 2021 Financial Results, Updates 2021 Financial Guidance

Scaling Phage Therapy

Tonix Pharmaceuticals Enrolls First Participant in the PRECISION Study, an Observational Study to Facilitate Development of Precision Medicine Techniques for COVID-19 Vaccines and Therapeutics

Synthetic Origins

FDA Returns Disappointing News for ALS Stem Cell Therapy

Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon Digest: Ghana Approves A Better Malaria Vaccine

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